Pharmaguy's Insights Into Drug Industry News
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Pharmaguy's Insights Into Drug Industry News
Pharmaguy curates and provides insights into selected drug industry news and issues.
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Patient Advocates Should Have Fought For Affordable Duchenne Drug as More Insurers Deny Coverage

Patient Advocates Should Have Fought For Affordable Duchenne Drug as More Insurers Deny Coverage | Pharmaguy's Insights Into Drug Industry News |

The story of Exondys 51 raises complex and emotionally charged questions about what happens when the F.D.A. approves an expensive drug based on a lower bar of proof. In practice, health insurers have taken over as gatekeeper in determining who will get the drug.


Disputes like the one over the Duchenne drug are likely to become more commonplace in the coming months. A federal law, passed last year, directs the agency to remove barriers to approving drugs and medical devices, and its new commissioner, Dr. Scott Gottlieb, has called on the F.D.A. to be more lenient, especially when it comes to rare pediatric diseases.


While insurers once covered drugs for rare diseases as a matter of course, that may be changing now that a wave of expensive drugs have reached the market. The pharmaceutical industry has been in hot pursuit of an increasingly enticing demographic target: An estimated 30 million people in the United States — about 10 percent of the population — are living with one of roughly 7,000 rare diseases.


The agency’s approval of Exondys 51, though, prompted a rebellion among some insurers, who are refusing to play along and saying they are concerned about the cumulative impact of such breathtakingly expensive drugs on health care costs. Anthem, one of the nation’s largest insurers, calls Exondys 51 “investigational” because the F.D.A. reserved the right to withdraw it from the market if future clinical trials fail to show it works.


Another insurer, Premera Blue Cross, went so far as to tie coverage to an invasive procedure — a muscle biopsy — but then rescinded the requirement.


“I’m reading a lot of denial letters,” said Christine McSherry, who until recently served as executive director of the Jett Foundation, an advocacy group that guides families through the insurance appeal process. Her insurer, Blue Cross Blue Shield of Massachusetts, is covering the drug for her son, Jett, through next April. “It’s very disheartening to have worked that hard, and to have sacrificed that much, and to now have to battle the insurance companies.”


The drug’s high cost is driving the resistance. While the drug manufacturer, Sarepta, has said Exondys 51 costs about $300,000 a year per child, the price, based on a child’s weight, can be much higher. For the dozen boys in the main clinical trial, the average list price would be more than double Sarepta’s quote — $750,000 each, according to an analysis by the drug benefit firm Prime Therapeutics.


“I think a lot of the advocates in this space maybe thought that getting a drug on the market was the goal of their advocacy,” said Dr. Aaron S. Kesselheim, an associate professor of medicine at Harvard University who voted against the drug’s approval as part of an F.D.A. advisory committee. “The goal of the advocacy should have been getting a product on the market, and making sure that it’s available at a reasonable cost.”


Further Reading:

  • “2nd Largest Health Insurer – Anthem – Won’t Pay for FDA-Approved Duchenne Drug”;
  • “FDA’s Approval of Exondys 51 for DMD Was Primarily Based on Money, Not Efficacy”;
Pharma Guy's insight:

This article also points out that this may be a trend for expensive drugs that are approved for rare diseases: "While insurers once covered drugs for rare diseases as a matter of course, that may be changing now that a wave of expensive drugs have reached the market. The pharmaceutical industry has been in hot pursuit of an increasingly enticing demographic target: An estimated 30 million people in the United States — about 10 percent of the population — are living with one of roughly 7,000 rare diseases."

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Pharma reputation from a patient perspective is growing! See the latest results of 2015 survey.

Pharma reputation from a patient perspective is growing! See the latest results of 2015 survey. | Pharmaguy's Insights Into Drug Industry News |

Reports published in 2016 on the Corporate Reputation of Pharma, as viewed by over 1,000 patient groups


The corporate Reputation of Pharma is growing:
In 2011 there was 42.0% of patient groups stating that the corporate reputation of the Pharma industry is "excellent" or "good". Although this percentage decreased to a 34% in 2012, it has increased ever since to 44.7% in 2015!

For a quick summary about our methodology and series of reports on corporate reputation of pharma and medical devices view the powerpoint here


Of course, this global indicators must be seen from the differentiated perspectives of regions and diseases!


Reports of Pharma's Corporate Reputation over regions:



Reports of Pharma's Corporate Reputation over therapeutic areas.:

Cancer; Circulatory conditions; Diabetes; HIV/Aids; Hepatitis; Neurological conditions; Mental health; Skin; Respiratory and Rare diseases.
Reports for Respiratory and Circulatory conditions are forthcoming. 

Via rob halkes
Pharma Guy's insight:

Related article: “Pharma's Rep Among Patient Groups at 4-Year High”; It should be noted that several of the patient organizations participating in this survey receive funding from the pharmaceutical industry. Also read, “#Pharma to Patient Advocacy Groups Questioning High Drug Prices: ‘Why Are You Doing This to Us?’”; and “Americans Hate the #Pharma Industry Almost as Much as They Hate U.S. Gov't!”;

rob halkes's curator insight, October 18, 2016 5:50 AM

Great to seen how pharma's Corporate Reputation in the eyes of the patients is growing globally from 2011 to 2015, with a dip in 2012. Study the nuances for different global regions and Disease conditions!

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Gang-Stalking - A Rare Disease Ripe for #Pharma Orphan Drug Solution?

Gang-Stalking - A Rare Disease Ripe for #Pharma Orphan Drug Solution? | Pharmaguy's Insights Into Drug Industry News |

Nobody believed him. His family told him to get help. But Timothy Trespas, an out-of-work recording engineer in his early 40s, was sure he was being stalked, and not by just one person, but dozens of them.


He would see the operatives, he said, disguised as ordinary people, lurking around his Midtown Manhattan neighborhood. Sometimes they bumped into him and whispered nonsense into his ear, he said.


“Now you see how it works,” they would say.


At first, Mr. Trespas wondered if it was all in his head. Then he encountered a large community of like-minded people on the internet who call themselves “targeted individuals,” or T.I.s, who described going through precisely the same thing.


The group was organized around the conviction that its members are victims of a sprawling conspiracy to harass thousands of everyday Americans with mind-control weapons and armies of so-called gang stalkers. The goal, as one gang-stalking website put it, is “to destroy every aspect of a targeted individual’s life.”


Mental health professionals say the narrative has taken hold among a group of people experiencing psychotic symptoms that have troubled the human mind since time immemorial. Except now victims are connecting on the internet, organizing and defying medical explanations for what’s happening to them.


The community of targeted individuals, conservatively estimated to exceed 10,000 members, has proliferated since 9/11, cradled by the internet and fed by genuine concerns over government surveillance. A large number appear to have delusional disorder or schizophrenia, psychiatrists say.


Yet, the phenomenon remains virtually unresearched.


For the few specialists who have looked closely, targeted individuals represent an alarming development in the history of mental illness: thousands of sick people, banded together and demanding recognition on the basis of shared paranoias.


They raise money, hold awareness campaigns, host international conferences and fight for their causes in courts and legislatures.

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"Venture Philanthropists" Bet Big on Orphan Drugs for Rare Diseases

"Venture Philanthropists" Bet Big on Orphan Drugs for Rare Diseases | Pharmaguy's Insights Into Drug Industry News |
Venture philanthropy risks benefiting companies, not patients.

ROBERT J. BEALL, the president and chief executive of the Cystic Fibrosis Foundation, called his recent decision to sell the royalty rights to his organization’s research a “game changer.” Indeed: Deals like this, in which an investment company paid the foundation $3.3 billion for its future royalties from several cystic fibrosis drugs it helped finance, could revolutionize the way medical research is funded. Rather than the staid model of government-funded institutions handing out grants to academic research facilities, a new breed of “venture philanthropies” like the Cystic Fibrosis Foundation could corral private investment into developing lifesaving drugs quickly and cheaply.

The problem is that venture philanthropy is, essentially, another term for privatizing scientific research. Instead of decisions about the fate of scientific funding being made by publicly oriented institutions, those decisions are being put in the hands of anonymous philanthropists and ostensibly benevolent nonprofits.

So far, there is no effort to extend government price controls to venture-philanthropy-derived research. The Cystic Fibrosis Foundation did little to lobby for lower prices on the drugs that were developed from the research it funded. As a result, Kalydeco, a cystic fibrosis medication it funded, is one of the most expensive drugs available, at $300,000 a year.

One argument in favor of venture philanthropy is that it creates a way to sustain small foundations that study rare diseases that, from a for-profit point of view, aren’t worth investigating.

But while Big Pharma might be faulted for funneling billions of dollars into erectile-dysfunction drugs and off-label drug marketing, researching extremely rare diseases may also represent a misuse of public and private funds. Efforts to cure, rather than treat or prevent, obscure diseases can be expensive, diverting investment from more common afflictions. The high costs of focusing on rare diseases are then eventually pushed onto the health care system by way of egregiously high drug prices. Such a choice involves an incredibly complex moral calculus, one that is best processed by democratic public institutions.

To make medical advancements truly philanthropic, the profit motive needs to be removed from the equation. If the intent is to cure rare diseases, then we should be increasing the budget for the National Institutes of Health and other research initiatives. Instead of gala balls and donor drives, higher taxes on the same rich benefactors could be used to fund the research that isn’t already being supported. Biotech patents developed through venture philanthropy should not have exclusive rights attached to them.

This would allow generic versions of drugs onto the market, which would go a long way toward keeping health care costs down and not driving the uninsured into debt.

Pharma Guy's insight:

When a dumb reporter asked "Willie" Sutton, the famous bank robber, why he robbed banks, he is reported to have said "Because that's where the money is." Duh!

Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an "orphan drug" -- ie, a drug for a disorder affecting fewer than 200,000 people in the U.S. -- you would likely have gotten a response such as "because there is an unmet medical need" or something similar.

Today, however, orphan drugs also have the potential to turn into blockbusters; ie, be where pharma's money is at (see "Analyst says smart money is on drugs for certain orphan diseases" and "New Big Pharma Economies of Scale: Less Patients Needed to Reach Blockbuster Sales").

From a Feb 5, 2015, editorial by a CF patient in Cystic Fibrosis Today:

Despite the incredible success of the CF Foundation in the sale of its royalty rights through the venture philanthropy model, concerns have been raised about collaboration between non-profits and pharmaceuticals.

“There is some concern that a profit motive could divert the organizations from their primary mission — helping patients — and create a conflict of interest,” Andrew Pollack comments in his New York Times article. “For instance, the price of the main drug developed through Cystic Fibrosis Foundation’s investment is $300,000 a year.”

The article addresses a serious issue surrounding the high cost of CF modulators that can be cost prohibitive for patients. The high costs begin to enter the ethical dilemma, which asks, how much would you pay to be healthy?

The argument presented in the article is that if non-profits such as the CF Foundation are receiving royalties from the sale of drugs, there is less incentive for them to lobby the company to reduce the costs for the patients. However, Dr. Beall of the CF Foundation has contended that despite their concerns for the high priced ivacaftor (kayldeco) there was little they could do to reduce the cost, which was at the discretion of Vertex.

I am the first to argue that it is an imperative that every person with CF who is eligible for novel CF modulator therapies have access to the drugs. However, it is misplaced to discredit the venture philanthropy model as a result, since the discussion surrounding the affordability of the drug only exists because the CF Foundation invested the capital to have it created.

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#Pharma Turning Patients With Rare Diseases Into D.C. Lobbyists

#Pharma Turning Patients With Rare Diseases Into D.C. Lobbyists | Pharmaguy's Insights Into Drug Industry News |

The pharmaceutical industry is teaming up with advocacy groups that are training and even paying for patients who need their medicines to promote their causes in Washington.


National polls identify high drug prices as Americans’ No. 1 health care complaint (read, for example, “Kaiser Health Tracking Poll: Americans Weigh In on How to Keep Drug Costs Down”;,  and President Trump has declared that pharmaceutical companies are “getting away with murder”; 


But these behind-the-scenes partnerships between the pharmaceutical industry and advocacy groups may work against reducing the price of high-price drugs. Critics say vulnerable patients are being manipulated and the goals promoted are skewed by the pharma benefactors who want faster government approval for new products and to get insurers or government programs to pay for them, whatever the price tag.


Dr. Ezekiel Emanuel, a bioethicist who has studied the issue, said he questions whether patient advocacy groups truly are “white knights defending the good fight.” He said research suggests that the conflicts of interest that occur when drug companies train and finance patient groups are “pretty rampant.” Emanuel co-authored a March study that found 83 percent of the 104 largest patient advocacy groups take money from the drug, medical device, and biotech industries (read "83% of Patient-Advocacy Organizations Receive Substantial Financial Support from the Drug Industry But Few Disclose How Much"). Smaller organizations are even more likely to be disproportionately dependent on industry funding for their operating budgets, he said.


The patient-lobbying conference, organized by the EveryLife Foundation for Rare Diseases, underlines how the financial interests of manufacturers and the medical needs of patients are intertwined.


More than 300 patients and advocates attended, and nearly all took part in a subsequent “Lobby Day” to visit congressional staff and lawmakers. They permitted a reporter from Kaiser Health News to observe and also join in a reception showcasing art by some of the patients.


Many patients were visibly sick or terminally ill. When deployed to pay visits to politicians, they add a human face to lobbying efforts around proposed legislation that affects pharma. Legislation like the Cures Act might increase spending on drug development or grease the pathway of drugs to market and with fewer regulations.

Before going to Capitol Hill, the patients and their families underwent a day of training, learning how to tell their stories. If at a loss for what to talk about, they were provided talking points on what EveryLife staffers called potential “asks.”


The group’s president, Dr. Emil Kakkis, is a drug industry executive. He said the foundation doesn’t “tell patients what to do on the Hill. They are given options.”


During one session called “Tricks of the Trade: Preparing for a Successful Meeting,” Soapbox Consulting chief executive Christopher Kush walked the audience through logistics for the next day.


The attendees were given a mobile app, which shows each advocate’s prearranged meeting list. Checking a map, Kush looked at the audience and said: “If you see a little dot where you live, you may have a new member of Congress—or a green check on your state, that means you have a new senator.”


Emanuel said he believes that patient advocacy groups should openly state their potential conflicts while participating in regulatory meetings. In addition, Emanuel said, drug and device manufacturers should annually report how much they pay patient advocacy groups just as they do with physicians and teaching hospitals.


Further Reading:

  • “PhRMA Deploys Scientists & Patients as Lobbyists on Capitol Hill”;
  • “How a #pharma Funded ‘Grassroots’ Patient Advocacy Campaign Changed FDA's Approval Process”;
  • “Pharma Lines Up Patient Groups to Fight for PDUFA Boondoggle”;
  • “The Ying Yang of Patient Advocate Groups and the Pharma Industry”;
  • “Transparency is Good in Theory, But Not in Practice”;
  • “#Pharma to Patient Advocacy Groups Questioning High Drug Prices: ‘Why Are You Doing This to Us?’";
  • “Is There No End to Mylan's Shenanigans? Paying Off Patient Groups to Lobby!”;
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Make American Medicine Great Again: #Pharma Should Offer Payers "Money Back Guarantees" as in UK

Make American Medicine Great Again: #Pharma Should Offer Payers "Money Back Guarantees" as in UK | Pharmaguy's Insights Into Drug Industry News |

Drug companies have started offering 'money back guarantees' in their treatments to make eye-wateringly expensive therapies more attractive.


This week British pharmaceutical giant GlaxoSmithKline (GSK) said it would include a warranty for its cutting-edge new gene therapy for ‘Bubble Boy syndrome’, a rare disorder which leaves suffers with such a compromised immune system that they are advised to live in a completely sterile environment.


GSK gained European approval for gene therapy Strimvelis in May but healthcare providers have been reluctant to pay for it, as it costs around £500,000 per patient, making it one of the most expensive treatments in the world.


The deal is thought to represent a new era in healthcare pricing, where drug companies will be responsible for continued success rates and allows treatments for very rare diseases, with low take-up, to come to market.


Such ‘money-back’ deals have only been made a four times in Britain in the past. The first was approved in 2002 for the treatment of multiple sclerosis with interferon beta or glatiramer where drug prices were adjusted according to outcomes.


In 2007 the NHS agreed a risk-sharing scheme with Johnson & Johnson after the National Institute for Health and Care Excellence (Nice) rejected its cancer drug Velcade.


Under the terms if a patient achieved complete or partial response, greater than 50 per cent within four cycles or treatment then the NHS would pay up. If not Johnson & Johnson agreed to pick up the entire bill.


Since then Merck Serono has reimbursed the health service for patients who failed to respond to its metastatic colorectal cancer drug Erbitux within six weeks. And GSK has a refund deal with its drug Votrient for Adcanced renal cell carcinoma.

Pharma Guy's insight:

I assume that in the U.S. scenario, patients would also get back their co-pays. Not that this would help patients who have died (like in this Opdivo case: But at lest insult would not be added to injury.

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FDA's Priority Review Voucher Program is Broken

FDA's Priority Review Voucher Program is Broken | Pharmaguy's Insights Into Drug Industry News |

There isn’t much profit in drugs or vaccines targeting rare diseases, especially when those rare diseases afflict people who don’t have a lot of money.

Since 2007, the US government has attempted to draw companies into this sparsely occupied space by creating an incentive program. Manufacturers who license products for specified “neglected” diseases are granted the right to an expedited Food and Drug Administration review when they want to bring another product to market.

It seems like a good idea — and may still be. But a number of groups that supported the program aren’t happy with how it is playing out so far.

The program is called the Priority Review Voucher Program for Neglected Diseases. When it was created, it covered specific diseases — among them, leprosy, malaria, and schistosomiasis and onchocerciasis, known as river blindness.

The vouchers don’t have to be used by the companies that earn them; they can be sold. Originally they could only be sold once. Last year that rule was changed and now a company that buys a voucher can resell it at a later date.

David Ridley and some colleagues at Duke University’s Fuqua Business School dreamed up the priority vouchers scheme — they published an articleproposing it in Health Affairs in 2006. Ridley always hoped the vouchers would earn big money for companies that receive them. And they have.

The first sold for $67.5 million. The next went for almost double that, $125 million. Then $245 million. In August, United Therapeutics sold a priority review voucher to Abbvie for $350 million. The others remain unused and if they have been sold, it hasn’t yet been disclosed.

But more vouchers mean there are more drugs for neglected diseases, right?

Not really. And that’s the problem.

Some of the vouchers have been awarded to drugs that have been on the market in other parts of the world for years, but have just been put through the licensure process in the United States. In fact, the first voucher was awarded to Novartis in 2009 for a combination malaria drug that had been licensed outside the US since 2001 and was already widely in use.

“This was not in any way an actual new treatment,” said Rachel Cohen, regional executive director for the North American division of the Drugs For Neglected Diseases Initiative. She noted the Rare Pediatric Diseases program does require that a treatment is novel in order to earn a voucher.

Another problem: Drug makers that earn priority review vouchers don’t have to guarantee that the drugs will actually be available, or sold at an affordable price. Janssen was awarded a voucher for Sirturo, used to treat multidrug resistant tuberculosis. Cohen said the drug is prohibitively priced in many parts of the world.

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NIH funds research consortia to study more than 200 rare diseases. Where's #Pharma?

NIH funds research consortia to study more than 200 rare diseases. Where's #Pharma? | Pharmaguy's Insights Into Drug Industry News |

Physician scientists at 22 consortia will collaborate with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases. The collaborations are made possible through awards by the National Institutes of Health --  totaling about $29 million in fiscal year 2014 funding -- to expand the Rare Diseases Clinical Research Network (RDCRN), which is led by NIH's National Center for Advancing Translational Sciences (NCATS). 

There are several thousand rare diseases, of which only a few hundred have any treatments available. Combined, rare diseases affect an estimated 25 million Americans. Some obstacles to developing rare disease treatments include difficulties in diagnosis, widely dispersed patients and scientific experts, a perception of high risk, and a lack of data from natural history studies, which follow a group of people with a specific medical condition over time. 

"NCATS seeks to tackle these challenges in an integrated way by working to identify common elements among rare diseases," said NCATS Director Christopher P. Austin, M.D. "The RDCRN consortia provide a robust data source that enables scientists to better understand and share these commonalities, ultimately allowing us to accelerate the development of new approaches for diagnosing and treating rare diseases." 

Many patients with rare diseases often struggle to obtain an accurate diagnosis and find the right treatments. In numerous cases, RDCRN consortia have become centers of excellence for diagnosing and monitoring diseases that few clinicians see on a regular basis.

Pharma Guy's insight:

An interesting Shire Pharmaceutical survey compares the health, psycho-social, and economic impact of rare diseases on patient and medical communities in the United States (US) and United Kingdom (UK). According to patients surveyed, it takes on average 7.6 years in the US for a patient with a rare disease to receive a proper diagnosis, whereas it takes 5.6 years in the UK. For more on that, read Rare Disease Sufferers in US Worse Off Financially & Medically Than Those in UK

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